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Our Pipeline

Circular photo of two Orna scientists in the lab, encircled in a red-to-purple ombre paintstroke

A Full-Circle Approach To
Treating Disease Paint Underline

We believe our oRNA™ and LNP technology can address the limitations of linear mRNA therapeutics and treat challenging diseases where other modalities have fallen short. With higher protein expression, larger cargo capacity, and lower immunogenicity profile, our therapeutics can go the distance and fill a pipeline with far-reaching effects in oncology, genetic disorders and infectious disease.

Pipeline Paint Underline

Product
Indication
Discovery
Preclinical
Clinical
Partners
isCARTM
Anti-CD19 CAR
B-cell lymphomas
ORN-101
Undisclosed
—

Orna’s in situ CAR therapy (isCAR™) combines oRNA™ technology with our proprietary LNPs to create modified immune cells within a patient, no lymphodepletion required. Unlike existing ex vivo CAR-T therapies, our isCAR™ program generates CAR-T, CAR-NK and CAR-Mac cells at once, and is truly off-the-shelf. This easily redosable format allows for reliable dose control and consistent generation of CAR-bearing immune cells for effective tumor control.

Through our partnership with Simnova Biotherapeutics, we are working to develop the China market for a number of multiple potential therapeutics in the area of oncology to deliver on the promise of circular RNA therapies.
Genetic Disease
Dystrophin
replacement
DMD
ReNAgade Therapeutics logo
Undisclosed
—
The large cargo capacity of oRNA™ allows for non-viral delivery of full-length protein-coding RNA in human cells to overcome a significant limitation in gene therapy. Combined with our second LNP delivery solution that targets liver and muscle, Orna is leveraging the vast potential of circular RNA in numerous areas.
Infectious Disease
Undisclosed
—
Merck logo
ReNAgade Therapeutics logo
RNA technology is ideal for vaccine development, as shown by the recent success of multiple mRNA-based vaccine products. In collaboration with Merck, Orna is leveraging the potency and durability of our oRNA™ platform coupled with novel LNP delivery insights from ourselves and our partner ReNAgade to develop the next generation of vaccine candidates.
Discovery Programs
Undisclosed
—
Orna is continuing to investigate new disease areas where oRNA™ and LNP technologies can address unmet need. Our enhanced platform capabilities guide our discovery program work.
isCARTM
Product: Anti-CD19 CAR
Indication: B-cell lymphomas
ORN-101
Discovery
Product: Undisclosed
Indication: —
Discovery

Orna’s in situ CAR therapy (isCAR™) combines oRNA™ technology with our proprietary LNPs to create modified immune cells within a patient, no lymphodepletion required. Unlike existing ex vivo CAR-T therapies, our isCAR™ program generates CAR-T, CAR-NK and CAR-Mac cells at once, and is truly off-the-shelf. This easily redosable format allows for reliable dose control and consistent generation of CAR-bearing immune cells for effective tumor control.

Through our partnership with Simnova Biotherapeutics, we are working to develop the China market for a number of multiple potential therapeutics in the area of oncology to deliver on the promise of circular RNA therapies.
Genetic Disease
Product: Dystrophin replacement
Indication: DMD
Partners:
ReNAgade Therapeutics logo
Discovery
Product: Undisclosed
Indication: —
Discovery
The large cargo capacity of oRNA™ allows for non-viral delivery of full-length protein-coding RNA in human cells to overcome a significant limitation in gene therapy. Combined with our second LNP delivery solution that targets liver and muscle, Orna is leveraging the vast potential of circular RNA in numerous areas.
Infectious Disease
Product: Undisclosed
Indication: —
Partners:
Merck logo
ReNAgade Therapeutics logo
Discovery
RNA technology is ideal for vaccine development, as shown by the recent success of multiple mRNA-based vaccine products. In collaboration with Merck, Orna is leveraging the potency and durability of our oRNA™ platform coupled with novel LNP delivery insights from ourselves and our partner ReNAgade to develop the next generation of vaccine candidates.
Discovery Programs
Product: Undisclosed
Indication: —
Discovery
Orna is continuing to investigate new disease areas where oRNA™ and LNP technologies can address unmet need. Our enhanced platform capabilities guide our discovery program work.